Compassionate Use of Defibrotide for Patients With Veno-occlusive Disease of the Liver

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

NO_LONGER_AVAILABLE

Study Classification

EXPANDED_ACCESS

Study Start Date

2003-11-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

Veno-occlusive disease (VOD) of the liver is a significant complication for some patients undergoing hematopoietic stem cell transplantation. This disease is thought to be a toxicity secondary to chemotherapy or radiation-induced damage to the epithelial cells of the blood vessels in the liver. VOD is categorized as mild, moderate or severe. Historically, there has been no method to treat the disease. Recently, however, there have been investigations into the use of a new agent called defibrotide.

The primary purpose of this protocol is to provide defibrotide to patients with severe VOD. Because this drug has not been approved by the FDA, use of this medication under the auspices of this IND treatment plan is for compassionate use only.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Pediatric Trial Investigating the Incidence & Outcome of Veno-Occlusive Disease With the Prophylactic Use of Defibrotide

NCT00272948

Hepatic Veno-Occlusive Disease

COMPLETED PHASE2/PHASE3

Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease in Hematopoetic Stem Cell Transplant Patients

NCT00358501

Severe Hepatic Veno-Occlusive Disease

COMPLETED PHASE3

An Open Study to Investigate the Safety and Efficacy of Replenine®-VF in Severe Haemophilia B Patients

NCT02231944

Haemophilia B

COMPLETED PHASE3

Liver Biopsy Following Gene Therapy For Hemophilia

NCT05932914

Hemophilia A Hemophilia B

NOT_YET_RECRUITING

Phase 1 Safety, Pharmacokinetics And Pharmacodynamics Study Of Recombinant Factor VIIa Variant (813d) In Adult Subjects With Hemophilia

NCT01439971

Hemophilia A

COMPLETED PHASE1

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Secondary objectives of this protocol include the following:

* To describe the toxicities of defibrotide in patients with VOD.
* To describe the response rate of VOD in patients receiving defibrotide.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Hepatic Veno-occlusive Disease

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Defibrotide

Defibrotide to be administered approximately every 6 hours at an initial dose of 6.25 mg/kg/dose. Subsequent doses will be escalated with a plateau of 15 mg/kg/dose (or 60 mg/kg/day total). For patients with persistent VOD, the dose may be escalated by 10 mg/kg/day as needed to a maximum of 110 mg/kg/day total.

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Veno-occlusive disease VOD Hepatic veno-occlusive disease.

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

Patients with clinical or pathologic diagnosis of veno-occlusive disease. The following criteria will be used:

1. Hyperbilirubinemia (greater than or equal to 2mg/dl) and two of the following: (hepatomegaly, ascites, or weight gain \[greater than or equal to 5% of baseline\]); OR,
2. Patient who do not fulfill criteria in #1 but who have pathologic or radiographic evidence of VOD.

Eligible Sex

ALL

Accepts Healthy Volunteers

No